211 top medical experts on GM1 Gangliosidosis across 23 countries and 17 U.S. states, including 51 MDs (Physicians). This is based on an objective analysis of their Scientific Publications, Clinical Trials, Medicare, and NIH Grants.

  1. GM1 Gangliosidosis: An autosomal recessive neurodegenerative disorder caused by the absence or deficiency of beta-galactosidase. It is characterized by intralysosomal accumulation of G(M1) ganglioside and oligosaccharides, primarily in neurons of the central nervous system. The infantile form is characterized by muscle hypotonia, poor psychomotor development, hirsutism, hepatosplenomegaly, and facial abnormalities. The juvenile form features hyperacusis; seizures; and psychomotor retardation. The adult form features progressive dementia; ataxia; and muscle spasticity. (From Menkes, Textbook of Child Neurology, 5th ed, pp96-7)
  2. Clinical guidelines are the recommended starting point to understand initial steps and current protocols in any disease or procedure:
  3. Broader Categories (#Experts): Gangliosidoses (66).
  4. Clinical Trials ClinicalTrials.gov : at least 16 including 3 Completed, 10 Recruiting
  5. Synonyms: G(M1) Gangliosidosis


  

        

                    


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